PDF(Pubmed)
Abstract：
UNASSIGNED: Autoimmune autonomic ganglionopathy (AAG) is characterized by serum autoantibodies against the ganglionic acetylcholine receptor (gAChR). Immunomodulatory treatments may alleviate AAG symptoms, but the most appropriate treatment strategy is unclear.
UNASSIGNED: This study aimed to confirm the effectiveness of treatments, particularly immunotherapy, in patients with seropositive AAG in Japan, as well as to determine the most effective treatment and the best assessment method for clinical response to treatment.
UNASSIGNED: We collected data from a previous cohort study of patients with seropositive AAG. The clinical autonomic and extra-autonomic symptoms were objectively counted and subjectively assessed using the modified Composite Autonomic Symptom Score. Post-treatment changes in the gAChR antibody level were evaluated.
UNASSIGNED: Thirty-one patients received immunotherapy. Among them, 19 patients received intravenous methylprednisolone; 27, intravenous immunoglobulin; 3, plasma exchange; 18, oral steroids; 2, tacrolimus; 1, cyclosporine; and 1, mycophenolate mofetil. Patients who received immunotherapy showed improvements in the total number of symptoms (from 6.2 ± 2.0 to 5.1 ± 2.0) and modified Composite Autonomic Symptom Score (from 37.4 ± 15.3 to 26.6 ± 12.8). Orthostatic intolerance, sicca, and gastrointestinal symptoms were ameliorated by immunotherapy. Immunotherapy decreased the antibody levels (gAChRα3 antibodies, from 2.2 ± 0.4 to 1.9 ± 0.4, p = 0.08; gAChRβ4 antibodies, from 1.6 ± 0.1 to 1.0 ± 0.2, p = 0.002), but antibody levels increased in 10 patients despite immunotherapy. The rate of improvement in the total number of symptoms was higher in patients with combined therapy than in patients with non-combined therapy (70.7% vs 28.6%).
UNASSIGNED: The scores in many items on the rating scale decreased after immunotherapy in patients with seropositive AAG, particularly in the combined immunotherapy group. However, more accurate assessment scales for clinical symptoms and multicenter randomized, placebo-controlled prospective studies are warranted to establish future treatment strategies.
UNASSIGNED: This study aimed to confirm the effectiveness of treatments, particularly immunotherapy, in patients with seropositive AAG in Japan, as well as to determine the most effective treatment and the best assessment method for clinical response to treatment.
UNASSIGNED: We collected data from a previous cohort study of patients with seropositive AAG. The clinical autonomic and extra-autonomic symptoms were objectively counted and subjectively assessed using the modified Composite Autonomic Symptom Score. Post-treatment changes in the gAChR antibody level were evaluated.
UNASSIGNED: Thirty-one patients received immunotherapy. Among them, 19 patients received intravenous methylprednisolone; 27, intravenous immunoglobulin; 3, plasma exchange; 18, oral steroids; 2, tacrolimus; 1, cyclosporine; and 1, mycophenolate mofetil. Patients who received immunotherapy showed improvements in the total number of symptoms (from 6.2 ± 2.0 to 5.1 ± 2.0) and modified Composite Autonomic Symptom Score (from 37.4 ± 15.3 to 26.6 ± 12.8). Orthostatic intolerance, sicca, and gastrointestinal symptoms were ameliorated by immunotherapy. Immunotherapy decreased the antibody levels (gAChRα3 antibodies, from 2.2 ± 0.4 to 1.9 ± 0.4, p = 0.08; gAChRβ4 antibodies, from 1.6 ± 0.1 to 1.0 ± 0.2, p = 0.002), but antibody levels increased in 10 patients despite immunotherapy. The rate of improvement in the total number of symptoms was higher in patients with combined therapy than in patients with non-combined therapy (70.7% vs 28.6%).
UNASSIGNED: The scores in many items on the rating scale decreased after immunotherapy in patients with seropositive AAG, particularly in the combined immunotherapy group. However, more accurate assessment scales for clinical symptoms and multicenter randomized, placebo-controlled prospective studies are warranted to establish future treatment strategies.
摘要：
■自身免疫性自主神经节病变（AAG）的特征是针对神经节乙酰胆碱受体（gAChR）的血清自身抗体。免疫调节治疗可以缓解AAG症状，但最合适的治疗策略尚不清楚.
■本研究旨在证实治疗的有效性，特别是免疫疗法，在日本血清AAG阳性的患者中，以及确定最有效的治疗方法和临床治疗反应的最佳评估方法。
■我们从先前AAG血清阳性患者的队列研究中收集数据。使用改良的复合自主神经症状评分对临床自主神经和自主神经外症状进行客观计数和主观评估。评估gAChR抗体水平的治疗后变化。
■31名患者接受了免疫治疗。其中,19例患者静脉注射甲基强的松龙；27例静脉注射免疫球蛋白；3例血浆置换；18例口服类固醇；2例他克莫司；1例环孢素；1例霉酚酸酯。接受免疫治疗的患者表现出症状总数的改善（从6.2±2.0到5.1±2.0）和改良的复合自主症状评分（从37.4±15.3到26.6±12.8）。直立不耐受,Sicca,胃肠道症状通过免疫疗法得到改善。免疫治疗降低了抗体水平(gAChRα3抗体，从2.2±0.4到1.9±0.4，p=0.08；gAChRβ4抗体，从1.6±0.1到1.0±0.2，p=0.002)，但10例患者的抗体水平增加，尽管免疫治疗。联合治疗患者的症状总数改善率高于非联合治疗患者（70.7％vs28.6％）。
■血清AAG阳性患者免疫疗法后，评定量表的许多项目得分均下降，特别是在联合免疫治疗组中。然而,更准确的临床症状评估量表和多中心随机，有必要进行安慰剂对照的前瞻性研究,以确定未来的治疗策略.
■本研究旨在证实治疗的有效性，特别是免疫疗法，在日本血清AAG阳性的患者中，以及确定最有效的治疗方法和临床治疗反应的最佳评估方法。
■我们从先前AAG血清阳性患者的队列研究中收集数据。使用改良的复合自主神经症状评分对临床自主神经和自主神经外症状进行客观计数和主观评估。评估gAChR抗体水平的治疗后变化。
■31名患者接受了免疫治疗。其中,19例患者静脉注射甲基强的松龙；27例静脉注射免疫球蛋白；3例血浆置换；18例口服类固醇；2例他克莫司；1例环孢素；1例霉酚酸酯。接受免疫治疗的患者表现出症状总数的改善（从6.2±2.0到5.1±2.0）和改良的复合自主症状评分（从37.4±15.3到26.6±12.8）。直立不耐受,Sicca,胃肠道症状通过免疫疗法得到改善。免疫治疗降低了抗体水平(gAChRα3抗体，从2.2±0.4到1.9±0.4，p=0.08；gAChRβ4抗体，从1.6±0.1到1.0±0.2，p=0.002)，但10例患者的抗体水平增加，尽管免疫治疗。联合治疗患者的症状总数改善率高于非联合治疗患者（70.7％vs28.6％）。
■血清AAG阳性患者免疫疗法后，评定量表的许多项目得分均下降，特别是在联合免疫治疗组中。然而,更准确的临床症状评估量表和多中心随机，有必要进行安慰剂对照的前瞻性研究,以确定未来的治疗策略.
